We in the Rare Disease Community Can Learn From Each Other

ALS is a life-changing diagnosis, and like many newly diagnosed patients, I was disappointed and frustrated at what felt like a standstill in medical progress against the disease. “If they can send humans into space, why can’t they cure ALS?” I’d lament, Now, 11 years later and with still…

Cytokinetics has awarded a total of $100,000 to five nonprofit patient advocacy organizations to support efforts to enhance communications, increase disease awareness, and boost community engagement. Recipients of the fourth annual Communications Fellowship Grant Program include the Les Turner ALS Foundation, the Northern Ohio Chapter of The…

Wave Life Sciences has gained additional financial support for an ongoing “basket” Phase 1b/2a clinical trial evaluating its experimental therapy, WVE-004, in people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene. So-called basket trials, commonly used in the field of cancer, test…

Eikonoklastes Therapeutics has added a candidate gene therapy for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases to its pipeline. The announcement follows the completion of a licensing agreement with the University of California San Diego. Brian Head, PhD, a professor with the university’s department of anesthesiology, and…

“Kristin, I need my eyes wiped again,” my husband, Todd, calls from his office. I had just put drops in his eyes 20 minutes ago, hoping to address the incessant itchiness he was experiencing, and now we are back at it again. I get up from my desk, grab a…

AI Therapeutics has launched a Phase 2 clinical trial into AIT-101 (apilimod dimesylate) as a potential oral treatment for amyotrophic lateral sclerosis (ALS) associated with C9orf72 mutations. The trial (NCT05163886) is currently recruiting at the Massachusetts General Hospital and is expected to soon open at sites in…

Amyotrophic lateral sclerosis (ALS) can be grouped into four disease subtypes based on patterns of changes in electrical signals in the brain, researchers have discovered. These findings may be valuable to predict future disability and life expectancy, and may help select the patients more likely to benefit in certain…

“‘There must be some way out of here,’ said the joker to the thief, ‘There’s too much confusion, I can’t get no relief.'” When I first heard “All Along the Watchtower” by Bob Dylan, I thought, with my 9-year-old sensibility, that the song referred to a monolith of timepieces.

Note: This story was updated Jan. 25, 2022, to clarify that Target ALS has raised $90 million since its inception in 2010. Amyotrophic lateral sclerosis (ALS) has been a part of Daniel Doctoroff’s life for more than two decades. His father, Martin Doctoroff, died of the disease in 2002, and…

Ady Barkan was a new father and activist when, at age 32, he learned that he had amyotrophic lateral sclerosis (ALS). His ensuing fight for universal healthcare while dealing with the progressive neurodegenerative disease is at the center of  “Not Going Quietly,” a film debuting on PBS today,…